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EP2906696A4 - Procédés pour moduler l'expression de c90rf72 - Google Patents

Procédés pour moduler l'expression de c90rf72

Info

Publication number
EP2906696A4
EP2906696A4 EP13846313.8A EP13846313A EP2906696A4 EP 2906696 A4 EP2906696 A4 EP 2906696A4 EP 13846313 A EP13846313 A EP 13846313A EP 2906696 A4 EP2906696 A4 EP 2906696A4
Authority
EP
European Patent Office
Prior art keywords
c90rf72
modulating
expression
methods
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
EP13846313.8A
Other languages
German (de)
English (en)
Other versions
EP2906696B1 (fr
EP2906696A1 (fr
EP2906696B2 (fr
Inventor
Frank Rigo
Don W Cleveland
Clotilde Lagier-Tourenne
John M Ravits
C Frank Bennett
Susan M Freier
Michael W Baughn
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ludwig Institute for Cancer Research Ltd
Ionis Pharmaceuticals Inc
University of California San Diego UCSD
Original Assignee
Ludwig Institute for Cancer Research Ltd
Ionis Pharmaceuticals Inc
University of California Berkeley
University of California San Diego UCSD
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=50488699&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=EP2906696(A4) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ludwig Institute for Cancer Research Ltd, Ionis Pharmaceuticals Inc, University of California Berkeley, University of California San Diego UCSD filed Critical Ludwig Institute for Cancer Research Ltd
Publication of EP2906696A1 publication Critical patent/EP2906696A1/fr
Publication of EP2906696A4 publication Critical patent/EP2906696A4/fr
Publication of EP2906696B1 publication Critical patent/EP2906696B1/fr
Application granted granted Critical
Publication of EP2906696B2 publication Critical patent/EP2906696B2/fr
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181Peptide nucleic acid, PNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Veterinary Medicine (AREA)
  • Psychiatry (AREA)
  • Public Health (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Liquid Crystal Substances (AREA)
EP13846313.8A 2012-10-15 2013-10-15 Procédés pour moduler l'expression de c90rf72 Active EP2906696B2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261714139P 2012-10-15 2012-10-15
PCT/US2013/065067 WO2014062686A1 (fr) 2012-10-15 2013-10-15 Procédés pour moduler l'expression de c90rf72

Publications (4)

Publication Number Publication Date
EP2906696A1 EP2906696A1 (fr) 2015-08-19
EP2906696A4 true EP2906696A4 (fr) 2016-06-08
EP2906696B1 EP2906696B1 (fr) 2019-10-09
EP2906696B2 EP2906696B2 (fr) 2022-12-14

Family

ID=50488699

Family Applications (1)

Application Number Title Priority Date Filing Date
EP13846313.8A Active EP2906696B2 (fr) 2012-10-15 2013-10-15 Procédés pour moduler l'expression de c90rf72

Country Status (7)

Country Link
US (1) US9963699B2 (fr)
EP (1) EP2906696B2 (fr)
DK (1) DK2906696T4 (fr)
ES (1) ES2762326T5 (fr)
FI (1) FI2906696T4 (fr)
PT (1) PT2906696T (fr)
WO (1) WO2014062686A1 (fr)

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EP3443094B1 (fr) * 2016-04-13 2022-10-19 Ionis Pharmaceuticals, Inc. Procédés de réduction de l'expression de c9orf72
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EP2906696A1 (fr) 2015-08-19
US20150267197A1 (en) 2015-09-24
DK2906696T4 (da) 2023-02-27
ES2762326T5 (es) 2023-04-27
DK2906696T3 (da) 2019-11-25
EP2906696B2 (fr) 2022-12-14
WO2014062686A1 (fr) 2014-04-24
ES2762326T3 (es) 2020-05-22
FI2906696T4 (fi) 2023-03-18
US9963699B2 (en) 2018-05-08
PT2906696T (pt) 2019-12-16

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